Contract to boost Medicaid access to sickle cell treatment
AIR's $28M CMS agreement focuses on cell and gene therapy medications
The American Institutes for Research (AIR) has been awarded a $28 million federal contract to help make cell and gene therapy (CGT) treatments more accessible to Medicaid recipients, initially focusing on sickle cell disease (SCD).
The contract, from the Center for Medicare and Medicaid Innovation in the Centers for Medicare and Medicaid Services (CMS), will support the implementation of the CGT Access Model, an approach in which CMS and states negotiate agreements based on outcomes.
“The Cell and Gene Therapy Access Model has the potential to make groundbreaking treatments more widely available to those who need them and reduce long-term healthcare costs,” Timothy Hill, AIR senior vice president and health division leader, said in an AIR press release.
SCD is a genetic disease that affects hemoglobin, the protein in red blood cells that carries oxygen throughout the body. The disorder affects more than 100,000 people in the U.S., most of whom are Black.
Red blood cells that contain the faulty version of hemoglobin adopt a sickle-like shape, which makes them more prone to getting trapped inside blood vessels and blocking blood flow. This, in turn, can lead to episodes of sudden and severe pain, known as vaso-occlusive crises.
CGT growing, but expensive
Cell and gene therapies are a growing class of one-time treatments that are designed to treat diseases such as SCD by addressing their underlying genetic cause.
Lyfgenia (lovotibeglogene autotemcel) is a one-time SCD gene therapy that aims to replace the defective gene encoding the faulty version of hemoglobin with a functional gene encoding a form of hemoglobin with anti-sickling properties. Casgevy (exagamglogene autotemcel) is a gene-editing therapy that’s designed to boost the production of fetal hemoglobin, a type of hemoglobin normally produced during early fetal development that transports oxygen more efficiently than its adult counterpart.
Despite these advances, such cell and gene therapies are costly and thus inaccessible to many patients on Medicaid, a U.S. government program that provides health insurance to low-income Americans. About half of SCD patients in the U.S. are Medicaid recipients.
The CGT Access Model will test whether an approach to negotiating and administering outcomes-based agreements led by states and the CMS, the agency that oversees Medicare, will improve access and outcomes for Medicaid recipients, reduce healthcare costs, and promote health equity. Under this type of agreement, the cost of a cell or gene therapy is based on how well it works for a specific patient.
AIR will monitor the administration and outcomes of the CGT Access Model and develop and enact a data collection and analysis strategy to assess health outcomes and financial indicators. AIR will also share its findings at conferences.
AIR’s principal researcher, Daniela Zapata, PhD, will direct the project. Partners include Deloitte and Wally Smith, MD, the Florence Neal Cooper Smith professor of SCD and director of the adult sickle cell program at Virginia Commonwealth University.
“We look forward to working with CMS and our partners to test innovative solutions that can improve people’s lives and create a more equitable world,” Hill said.
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